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Paper Details

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.
Front Immunol
7
2023
(AAV)- donor, -, - HSPCs, CRISPR, Cas9, HSPCs, IEIs, Inborn errors of, LV, LV- and TI- HSPCs, LV- or TI-treated XSCID HSPCs, NK cells, Pre-T-I, Pre-T-I and Pre-T-II lymphocytes, SCID-X1, X, X-linked Severe Combined Immunodeficiency, X-linked severe combined immunodeficiency, X-linked severe combined immunodeficiency hematopoietic stem cells, XSCID, XSCID HSPCs, autologous hematopoietic stem/progenitor cells, edited cells, endogenous locus, genome, mice, non-targeted vector integration sites, off-target sites, patients, rhAmpSeq, transgene
Author NameAffiliation
Sandra BurkettCenter for Cancer Research, National Cancer Institute, National Institutes of Health
Sandra BurkettCenter for Cancer Research, National Cancer Institute, National Institutes of Health
Benjamin P KleinstiverCenter for Genomic Medicine and Department of Pathology, Massachusetts General Hospital
Benjamin P KleinstiverHarvard Medical School
Xiaolin WuLeidos Biomedical Research Inc.
Xiaolin WuLeidos Biomedical Research Inc.
Matthew H PorteusStanford University
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